X4 Pharmaceuticals (XFOR) investor relations material

Executive summary

• Focused on developing and commercializing therapeutics for rare hematology diseases, with XOLREMDI (mavorixafor) as the lead product approved in the US and EU for WHIM syndrome, with commercialization in the EU led by Norgine.

• Progressing the pivotal Phase 3 4WARD trial for chronic neutropenia, with over 110 active sites, strengthened clinical operations via a top CRO, and ongoing FDA discussions.

• Completed transfer of EU marketing authorization for XOLREMDI to Norgine following regulatory approval.

• Cash runway projected into 2029, supporting ongoing clinical and commercial activities.

Financial highlights

• Total revenue for Q2 2026 was $8.8M, with $2.4M from US product sales and $6.4M from the Norgine license agreement.

• Net product sales grew to $2.4M in Q2 2026 from $1.7M in Q2 2025, driven by increased patient prescriptions.

• Net loss for Q2 2026 was $16.2M ($0.13 per share), improved from $25.7M ($3.47 per share) in Q2 2025.

• R&D expenses for Q2 2026 were $15.1M, down from $18.4M year-over-year, mainly due to reduced headcount post-2025 restructuring.

• Cash, cash equivalents, and marketable securities totaled $208.0M as of June 30, 2026; another report lists $195.1M.

Outlook and guidance

• Current cash and equivalents expected to fund operations and debt obligations for at least the next 12 months, with some projections extending the runway into 2029.

• Additional capital will be needed to fund operations beyond this period; future funding may come from equity, debt, or partnerships.

• Update on FDA meeting and 4WARD trial enrollment completion expected by end of Q3 2026.

• Potential to receive up to €221M in milestone payments and escalating double-digit royalties from Norgine for EU commercialization.

• Ongoing focus on completing the 4WARD trial and expanding XOLREMDI's market presence.